First human trial aims to treat rare brain disorder in kids
NCT ID NCT07600658
First seen May 23, 2026 · Last updated Jun 12, 2026 · Updated 4 times
Summary
This early-stage trial tests a new drug called TS1-ASO in 5 children with Timothy Syndrome, a rare genetic condition affecting the heart and brain. The drug is given via spinal injection to see if it is safe and can improve or prevent developmental and behavioral problems. The study focuses on safety and how the drug moves through the body, with no comparison group.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Stanford University
Stanford, California, 94305, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.